Pharmacological treatment of Duchenne and Becker muscular dystrophies

Currently, there is no cure for muscular dystrophy. Treatment is generally aimed at controlling the onset of symptoms to maximize the quality of life. Medical therapy contains exon-skipping agents, utrophin upregulation, histone deacetylase inhibitors, corticosteroids, myostatin blockers, ataluren, and tyrosinekinase inhibitors. Exon skipping uses antisense oligonucleotides as a treatment for genetic diseases. The antisense oligonucleotides used for exon skipping are designed to bypass premature stop codons in the target RNA and restore reading frame disruption. Utrophin upregulation results improved sarcolemmal stability and prevents dystrophic pathology through a significant reduction of regeneration, necrosis and fibrosis.egységes, osztatlanáltalános orvosango